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Flecainide for Catecholaminergic Polymorphic Ventricular Tachycardia

Not Applicable
Completed
Conditions
Catecholaminergic Polymorphic Ventricular Tachycardia
Interventions
Registration Number
NCT01117454
Lead Sponsor
Vanderbilt University Medical Center
Brief Summary

The purpose of this study is to test whether the addition of oral flecainide to standard therapy will reduce ventricular ectopy on exercise test compared to placebo plus standard therapy in patients with Catecholaminergic Polymorphic Ventricular Tachycardia.

Detailed Description

Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT) is a genetic arrhythmia syndrome characterized by frequent ventricular ectopy and polymorphic, classically bidirectional ventricular tachycardia with physical or emotional stress, which also carries a risk of ventricular fibrillation and sudden death, despite no structural heart abnormality. Treatment consists of beta-blockers and/or calcium channel blockers, but up to 30% of patients require implantable cardioverter-defibrillators (ICDs) due to recurrent symptoms on medical therapy. In an animal model, flecainide was found to directly target the molecular defect in CPVT. In a retrospective clinical study in patients with CPVT we have seen improvement of ventricular ectopy on exercise tests when flecainide is added to standard therapy. We propose a prospective trial of flecainide added to standard therapy in CPVT patients to test the hypothesis that flecainide will reduce ventricular ectopy on exercise testing compared to placebo plus standard therapy.

This will be a single-blind (blinded subjects) randomized cross-over study, in which each patient will receive treatment A (flecainide or placebo) for at least 3 months and, after a 1 week wash-out, treatment B (placebo or flecainide) for at least 3 months.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
14
Inclusion Criteria
  1. Clinical diagnosis of CPVT, based on:

    A. reproducible polymorphic or bidirectional ventricular tachycardia with exercise OR B. Ventricular ectopy on exercise test with RYR2 or CASQ2 mutation

  2. Functioning ICD in place

  3. On stable dose of standard therapy defined as the maximal tolerated dose of beta-blocker and may include a calcium channel blocker

Patients on flecainide or mexiletine are also eligible for enrollment after a 1 week "washout" period during which flecainide or mexiletine is discontinued, and standard therapy alone is used.

Exclusion Criteria
  1. Females who are pregnant or plan to be pregnant during the study period
  2. Children < 5 years of age
  3. Patients unable to perform treadmill exercise
  4. Patients with significant structural heart disease
  5. Patients with features consistent with Andersen-Tawil syndrome A. Periodic paralysis or unexplained weakness B. Dysmorphic facies C. Known KCNJ2 mutation
  6. Patients with known hypersensitivity to flecainide
  7. Patients on amiodarone
  8. Patients not expected to comply with follow-up

Study & Design

Study Type
INTERVENTIONAL
Study Design
CROSSOVER
Arm && Interventions
GroupInterventionDescription
Flecainide then placeboBeta blockerIn this crossover study, half of the subjects will be randomized to flecainide plus standard therapy with beta-blockers first, then crossover to placebo plus standard therapy with beta-blockers.
Flecainide then placeboPlaceboIn this crossover study, half of the subjects will be randomized to flecainide plus standard therapy with beta-blockers first, then crossover to placebo plus standard therapy with beta-blockers.
Placebo then flecainidePlaceboIn this crossover study, half of the subjects will be randomized to placebo plus standard therapy with beta-blockers first, then crossover to flecainide plus standard therapy with beta-blockers.
Placebo then flecainideBeta blockerIn this crossover study, half of the subjects will be randomized to placebo plus standard therapy with beta-blockers first, then crossover to flecainide plus standard therapy with beta-blockers.
Flecainide then placeboFlecainide AcetateIn this crossover study, half of the subjects will be randomized to flecainide plus standard therapy with beta-blockers first, then crossover to placebo plus standard therapy with beta-blockers.
Placebo then flecainideFlecainide AcetateIn this crossover study, half of the subjects will be randomized to placebo plus standard therapy with beta-blockers first, then crossover to flecainide plus standard therapy with beta-blockers.
Primary Outcome Measures
NameTimeMethod
Number of Patients With Ventricular Ectopy or VT During Exercise Treadmill Testing3 months

Hypothesis: the addition of oral flecainide to standard therapy will reduce ventricular ectopy and/or VT on treadmill exercise treadmill testing in patients with CPVT, compared to placebo plus standard therapy.

Secondary Outcome Measures
NameTimeMethod

Trial Locations

Locations (10)

University of California Los Angeles

🇺🇸

Los Angeles, California, United States

Nationwide Children's Hospital

🇺🇸

Columbus, Ohio, United States

MetroHealth Medical Center

🇺🇸

Cleveland, Ohio, United States

Vanderbilt University

🇺🇸

Nashville, Tennessee, United States

University of Utah

🇺🇸

Salt Lake City, Utah, United States

Children's Hospital of Orange County

🇺🇸

Orange, California, United States

NYU Langone Medical Center

🇺🇸

New York, New York, United States

Duke University

🇺🇸

Durham, North Carolina, United States

East Carolina University

🇺🇸

Greenville, North Carolina, United States

Cook Children's Hospital

🇺🇸

Fort Worth, Texas, United States

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